European Journal of Paediatric Neurology最新文献

{"title":"Cognitive function in SMA patients with 2 or 3 SMN2 copies treated with SMN-modifying or gene addition therapy during the first year of life","authors":"Paula Steffens ,&nbsp;Deike Weiss ,&nbsp;Anna Perez ,&nbsp;Manuel Appel ,&nbsp;Philipp Weber ,&nbsp;Claudia Weiss ,&nbsp;Corinna Stoltenburg ,&nbsp;Ute Ehinger ,&nbsp;Maja von der Hagen ,&nbsp;Jens Schallner ,&nbsp;Birte Claussen ,&nbsp;Ilka Lode ,&nbsp;Andreas Hahn ,&nbsp;Rahel Schuler ,&nbsp;Lena Ruß ,&nbsp;Andreas Ziegler ,&nbsp;Jonas Denecke ,&nbsp;Jessika Johannsen","doi":"10.1016/j.ejpn.2024.05.002","DOIUrl":"10.1016/j.ejpn.2024.05.002","url":null,"abstract":"<div><h3>Background</h3><p>Spinal muscular atrophy (SMA) is a neuromuscular disease, causing progressive muscle weakness due to loss of lower motoneurons. Since 2017, three therapies, two modifying gene transcription and one adding the defective gene, have been approved with comparable efficacy on motor outcome. Data on cognitive outcomes of treated SMA type 1 patients is limited. The aim of this study was to evaluate cognitive function in symptomatic and presymptomatic SMA type 1 patients with two or three <em>SMN2</em> copies who received SMN-modifying or gene-addition therapy in the first year of life.</p></div><div><h3>Methods</h3><p>Cognitive testing was performed in 20 patients, including 19 symptomatic SMA type 1 patients with up to three SMN2 copies and 1 pre-symptomatically treated patient. Children were tested using Bayley Scales of Infant Development (BSID-III) at the age of 2 or 3 years or the Wechsler Preschool and Primary Scale of Intelligence (WPSII-IV) at the of age of 5 years.</p></div><div><h3>Results</h3><p>11/20 patients showed subnormal cognitive development. Boys had significantly lower cognitive scores. Patients requiring assisted ventilation or feeding support were more likely to have cognitive deficits. Achieving more motor milestones was associated with a better cognitive outcome.</p></div><div><h3>Conclusion</h3><p>Treated patients with SMA type 1 have heterogeneous cognitive function with 55 % of patients showing deficits. Risk factors for cognitive impairment in our cohort were male gender and need for assisted ventilation or feeding support. Therefore, cognitive assessment should be included in the standard of care to allow early identification of deficits and potential therapeutic interventions.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"51 ","pages":"Pages 17-23"},"PeriodicalIF":3.1,"publicationDate":"2024-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1090379824000606/pdfft?md5=5e1814a093678023085fa10f43835981&pid=1-s2.0-S1090379824000606-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141049200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Insights into Rett Syndrome girls' receptive vocabulary through eye movements and parental perception","authors":"Jucineide S. Xavier ,&nbsp;Fernanda T. Orsati ,&nbsp;Paulo G. Laurence ,&nbsp;Lourenço K. Barbosa ,&nbsp;Andressa S. Gouveia ,&nbsp;Maria Elóisa F. D'Antino ,&nbsp;Katerina Lukasova ,&nbsp;Elizeu C. Macedo ,&nbsp;José Salomão Schwartzman","doi":"10.1016/j.ejpn.2024.05.001","DOIUrl":"10.1016/j.ejpn.2024.05.001","url":null,"abstract":"<div><h3>Objective</h3><p>This study evaluated the receptive vocabulary of girls diagnosed with Rett Syndrome (RS) by employing eye-tracking technology and examined how these objective measures compared with parents' perceptions of their daughters' language abilities.</p></div><div><h3>Method</h3><p>Fourteen girls with RS and eleven typically developing peers participated. Instruments included the Kerr Scale, a parental questionnaire on communication skills, the Peabody Picture Vocabulary Test - PPVT-4, and eye-tracking equipment. The PPVT-4's initial 12 cards, each displaying four images, were presented on screen for 5 s for the child's initial inspection. Subsequently, the same card was shown for 5 s with instructions to identify the picture matching the word, repeated three times in a randomized order.</p></div><div><h3>Results</h3><p>Eye-tracking assessments revealed that girls with RS with less severe symptoms, performed below typically developing peers with aged 3–4 years. The number of eye fixations emerged as a reliable metric for assessing their performance. A positive correlation was found between parental reports of their daughters' vocabulary knowledge and their ability to identify the correct stimulus, suggesting comprehension and active engagement in the testing process.</p></div><div><h3>Conclusion</h3><p>This study presents a pioneering approach by combining parental perceptions with eye-tracking measures to assess receptive vocabulary in girls diagnosed with Rett Syndrome. Despite showing lower performance levels compared to significantly younger peers, the findings suggest that these individuals use less effectively eye contact as a mode of communication.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"51 ","pages":"Pages 1-8"},"PeriodicalIF":3.1,"publicationDate":"2024-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140904505","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Commentary on Phase IV PROVE study: Perampanel in real-world clinical care of pediatric patients with epilepsy [Moretz K, Wheless J, Santos C, Segal E, Lancman M, Patten A, Malhotra M]","authors":"Frank M.C. Besag,&nbsp;Michael J. Vasey","doi":"10.1016/j.ejpn.2024.05.008","DOIUrl":"10.1016/j.ejpn.2024.05.008","url":null,"abstract":"","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Page vii"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141082842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Blood parameters in pediatric myelin oligodendrocyte glycoprotein antibody-associated disorders","authors":"Alina Peternell ,&nbsp;Christian Lechner ,&nbsp;Markus Breu ,&nbsp;Martin Preisel ,&nbsp;Mareike Schimmel ,&nbsp;Astrid Eisenkölbl ,&nbsp;Joachim Zobel ,&nbsp;Eva-Maria Wendel ,&nbsp;Markus Reindl ,&nbsp;Kevin Rostásy ,&nbsp;Matthias Baumann","doi":"10.1016/j.ejpn.2024.04.011","DOIUrl":"10.1016/j.ejpn.2024.04.011","url":null,"abstract":"<div><h3>Background and objectives</h3><p>Patients with myelin oligodendrocyte glycoprotein antibody-associated disorders (MOGAD) clinically present e.g. with acute disseminated encephalomyelitis (ADEM), optic neuritis (ON), transverse myelitis (TM) or aquaporin-4-IgG (AQP4-IgG) negative neuromyelitis optica spectrum disorders (NMOSD)-like phenotypes. We aimed to analyze and compare blood parameters in children with MOGAD, AQP4-IgG-positive NMOSD (hence NMOSD), multiple sclerosis (MS) and healthy controls (HC).</p></div><div><h3>Methods</h3><p>We evaluated differences in complete blood counts (CBC), neutrophil-to-lymphocyte ratio (NLR), platelet-to-lymphocyte ratio (PLR), monocyte-to-lymphocyte ratio (MLR) and C-reactive protein (CRP) between these four groups and within the groups between clinical attack, acute treatment and remission.</p></div><div><h3>Results</h3><p>Our cohort consisted of 174 children and adolescents with a total of 550 timepoints: 66 patients had MOGAD (202 timepoints), 11 NMOSD (76 timepoints), 58 MS (219 timepoints) and 39 were HC (53 timepoints). At clinical attack, leukocyte counts were elevated in MOGAD compared to remission (p &lt; 0.001) and compared to all other groups (p &lt; 0.001). NLR was high in MOGAD and NMOSD, and PLR was high in NMOSD, however, after correction for multiple testing these findings did not remain significant. While glucocorticoids caused an increase of leukocyte counts and NLR in NMOSD and MS, these values remained stable during acute treatment in MOGAD. In remission, NLR normalized in MOGAD, while it stayed high in NMOSD. PLR increased in NMOSD and was significantly higher compared to all other groups.</p></div><div><h3>Discussion</h3><p>Some blood parameters, mainly leukocyte and differential counts, might help clinicians to evaluate disease activity, differentiate relapses from pseudo-relapses and even distinguish between different disease entities.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages 86-95"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1090379824000576/pdfft?md5=c76d803f4359c49728209b43ab083a78&pid=1-s2.0-S1090379824000576-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140782794","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neurologic complications of thiamine (B1) deficiency following bariatric surgery in adolescents","authors":"Lital Cohen Vig ,&nbsp;Rachel Straussberg ,&nbsp;Noa Ziv ,&nbsp;Lior Hirschfeld-Dicker ,&nbsp;Osnat Konen ,&nbsp;Sharon Aharoni","doi":"10.1016/j.ejpn.2024.04.008","DOIUrl":"https://doi.org/10.1016/j.ejpn.2024.04.008","url":null,"abstract":"<div><h3>Background</h3><p>The prevalence of obesity among children and adolescents is rising and poses a major health concern. Bariatric surgery is well established in adults and has become an option for adolescents. Thiamine (B1) deficiency is common following bariatric surgery in adults. It may present as Beri-Beri, Wernicke encephalopathy, or Korsakoff psychosis.</p></div><div><h3>Objective</h3><p>Our aim was to describe the clinical features, diagnosis, and treatment of adolescents who presented with B1 deficiency after bariatric surgery at one center, and to summarize the data from the literature.</p></div><div><h3>Patients</h3><p>Three adolescents with morbid obesity (two boys and one girl, aged 15.5 to- 17-years-old), presented at Schneider Children's Medical Center of Israel with progressive lower limb pain and weakness 2–3 month following a bariatric procedure (sleeve gastrectomy or narrowing of a bariatric band). The girl also had upper limb involvement and cerebellar signs. All three were non-compliant with micronutrient supplementation. After admission, they received intravenous B1 and oral multivitamin supplementation, and their symptoms improved considerably.</p></div><div><h3>Conclusions</h3><p>Micronutrient supplementation following bariatric surgery is crucial to prevent deficiencies. In adolescents, compliance with micronutrient supplementation should be assessed before and after such surgery. Thiamine deficiency may cause polyneuropathy, among other symptoms. Treatment reduces the severity of neurological complications.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages 74-80"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140822528","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"“Deconstructing” upper limb function in dyskinetic cerebral palsy","authors":"Roser Pons,&nbsp;Dalivigka Zoi","doi":"10.1016/j.ejpn.2024.05.005","DOIUrl":"10.1016/j.ejpn.2024.05.005","url":null,"abstract":"","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages v-vi"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140960658","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical correlation between disease progression and central vein sign in pediatric onset multiple sclerosis: A binational study","authors":"Shay Menascu ,&nbsp;Simona Halusková ,&nbsp;Amir Pollak ,&nbsp;Pavel Ryska ,&nbsp;Francesco Angelucci ,&nbsp;David Magalashvili ,&nbsp;Diana Guber ,&nbsp;Arthur Yosef ,&nbsp;Alon Kalron ,&nbsp;Martin Valis ,&nbsp;Michael Gurevich","doi":"10.1016/j.ejpn.2024.04.007","DOIUrl":"10.1016/j.ejpn.2024.04.007","url":null,"abstract":"<div><h3>Background</h3><p>The central vein sign (CVS) has been proposed as a novel MRI biomarker to improve diagnosis of pediatric-onset MS (POMS). However, the role of CVS in POMS progression has yet to be discovered.</p></div><div><h3>Objectives</h3><p>To investigate the appearance of CVS and its correlation with POMS disease progression.</p></div><div><h3>Methods</h3><p>One hundred fifty-six POMS from two MS centers in Israel and Czech Republic MS centers were followed for five years. Patient assessment was performed by the Expanded Disability Status Scale (EDSS) and Annual Relapse Rate (ARR). Patients in whom at least 40 % of brain MRI lesions had CVS (“rule of 40”) were determined as CVS-positive.</p></div><div><h3>Results</h3><p>The total group of POMS consisted of 96 CVS-negative (61.5 %), aged 14.6 ± 1.9 years, EDSS 2.0, 75 % Interquartile Range (IQR) 1.0–3.0, disease duration (DD) 6.28 ± 0.38 years, and 60 CVS-positive (38.5 %), aged 15.1 ± 0.3 years, EDSS 2.0, IQR 1.5–3.0, DD 5.62 ± 0.13 years, were analyzed. After a three and five-year follow-up, the CVS-positive patients had higher EDSS scores than those who were CVS-negative, 2.0, IQR 1.0–2.5, vs 1.0, IQR 1.0–2.0, (p = 0.009) and 2.0, IQR 1.0–3.25 vs 1.0, IQR 1.0–2.0, (p = 0.0003), respectively. Patients with CVS-positive POMS were characterized by a significantly higher ARR (0.78 ± 0.08 vs 0.57 ± 0.04, p = 0.002). These results were confirmed in subgroups of Disease Modifying Treatments (DMT) untreated and treated patients.</p></div><div><h3>Conclusion</h3><p>CVS-positive POMS is characterized by higher disability progression than CVS-negative, indicating the importance of CVS in disease pathogenesis.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages 81-85"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140770979","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Quality of life and support needs in children, adolescents, and young adults with facioscapulohumeral dystrophy, a mixed-method study","authors":"Jildou N. Dijkstra ,&nbsp;Nathaniël B. Rasing ,&nbsp;Helena T.M. Boon ,&nbsp;Sandra Altena-Rensen ,&nbsp;Edith H.C. Cup ,&nbsp;Anke Lanser ,&nbsp;Ietske J. Siemann ,&nbsp;Baziel G. van Engelen ,&nbsp;Corrie E. Erasmus ,&nbsp;Nicol C. Voermans","doi":"10.1016/j.ejpn.2024.04.006","DOIUrl":"10.1016/j.ejpn.2024.04.006","url":null,"abstract":"<div><h3>Background and objectives</h3><p>Quality of life (QoL) in children with facioscapulohumeral dystrophy (FSHD) seems plausible decreased. Little is known about factors influencing QoL in children with FSHD. Our objective is to explore factors contributing to the QoL of children, adolescents, and young adults with FSHD, to describe how they experience life with FSHD, and to report their support needs.</p></div><div><h3>Methods</h3><p>We performed a mixed-method study with individual age-appropriate semi-structured interviews assessing QoL in children, adolescents, and young adults with FSHD and their parents. To characterize the sample, quantitative data on QoL, pain, fatigue, and participation were collected. Interview data was analyzed using a thematic analysis.</p></div><div><h3>Results</h3><p>Fourteen patients participated (age between 9 and 26 years old, eight males and six females). The degree of FSHD severity, as indicated by the FSHD-score, did not correlate with QoL. Older children had a lower QoL than younger children. Children and adolescents strived for normality regardless of physical discomfort. Phenotypical features of FSHD led to insecurity aggravated by hurtful comments of others. The unpredictability of disease progression and its implications for career and parenthood choices led to a generalized feeling of uncertainty about the future. Support was found within family and friends. Participants expressed a need for peer support and psychological support as well as recommending it to others.</p></div><div><h3>Discussion</h3><p>Quality of life in childhood FSHD is diminished caused by their physical limitations, altered appearance, fear of social rejection, and uncertainty of the disease progression in the future. A fear of social rejection most likely contributes to striving for normality regardless of physical discomfort. Support should be focused on acceptance and coping with hurtful comments. It should preferably be individualized, easily accessible and not offered as therapy but rather as tutoring for children.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages 64-73"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1090379824000527/pdfft?md5=2c52f9dd27a762560e049d9a2acbb84e&pid=1-s2.0-S1090379824000527-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140784748","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Frequency of an intrathecal IgM synthesis and MRZ reaction in children with MS","authors":"Sandra Bigi","doi":"10.1016/j.ejpn.2024.05.009","DOIUrl":"10.1016/j.ejpn.2024.05.009","url":null,"abstract":"","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Page i"},"PeriodicalIF":3.1,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141089110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Efficacy and safety of N-acetyl-L-leucine in patients with ataxia telangiectasia: A randomized, double-blind, placebo-controlled, crossover clinical trial","authors":"Mehran Beyraghi-Tousi ,&nbsp;Amirhosein Sahebkar ,&nbsp;Mahsa Houra ,&nbsp;Pooria Sarvghadi ,&nbsp;Tannaz Jamialahmadi ,&nbsp;Reza Bagheri ,&nbsp;Shima Tavallaie ,&nbsp;Eric Gumpricht ,&nbsp;Maryam Saberi-Karimian","doi":"10.1016/j.ejpn.2024.04.009","DOIUrl":"https://doi.org/10.1016/j.ejpn.2024.04.009","url":null,"abstract":"<div><h3>Background</h3><p>Ataxia telangiectasia (AT) is an autosomal recessive multisystem disorder. Most patients have progressive cerebellar ataxia, oculocutaneous telangiectasia, frequent pulmonary infection, and an increased risk of malignancies. Although N-acetyl-<span>dl</span>-leucine (ADLL) has shown some efficacy in patients with AT, its more pharmacologically active enantiomer, N-acetyl-<span>l</span>-leucine (NALL), has just recently been investigated in ataxic individuals. The current study assessed the efficacy of NALL in patients with AT.</p></div><div><h3>Methods</h3><p>This 2 × 2 crossover, double-blind, randomized clinical trial was conducted on 20 patients with AT. After excluding four patients, 16 subjects (eight females, eight males; mean age 9.8 ± 3.5 years) with a definitive genetic diagnosis of AT were randomly assigned to one of two study groups, with one group receiving 1–4 g/day NALL or a placebo for six weeks. Subjects then had a 4-week washout before crossing over to the other treatment for an additional six weeks. The Spinocerebellar Ataxia Functional Index (SCAFI) and the Scale for Assessment and Rating of Ataxia (SARA) score assessed patients' motor function. Quality of life (QOL) was evaluated by a specialist using the PedsQL questionnaire. Fasting blood samples were taken from all subjects before and after each intervention to determine potential side effects.</p></div><div><h3>Results</h3><p>Although patients' nausea and constipation were improved, the results failed to reveal any significant benefits of NALL treatment on ataxia symptoms. NALL treatment had no significant effects on SARA, SCAFI-9HPT (9-hole peg test) nondominant, SCAFI-9HPT dominant, or SCAFI-8WMT (8 m walking time) (p &gt; 0.05). Our patient's Physical Health score in Child self-report and Parent proxy-report did not significantly change in the treatment group compared to the placebo (p &gt; 0.05). Furthermore, there were no significant changes in energy and macronutrient intake after NALL treatment. None of the volunteers reported serious or moderate side effects.</p></div><div><h3>Conclusions</h3><p>To the best of our knowledge, this was the first placebo-controlled, randomized clinical trial exploring NALL's potential effects for treating AT. Despite improvements in some symptomss, NALL intervention failed to improve motor function significantly. However, patients' nausea and constipation were improved by NALL, which can be a relevant benefit clinically.</p></div>","PeriodicalId":50481,"journal":{"name":"European Journal of Paediatric Neurology","volume":"50 ","pages":"Pages 57-63"},"PeriodicalIF":3.1,"publicationDate":"2024-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140647578","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}